Entrepreneurship and SMEs

Innovation

Health Care

Horizon2020

Research

Clinical trials

Specific Challenge:

On average it takes about 14 years for a new drug to travel from the research lab to market approval at an average cost of ≥€2 billion. Only 10% of compounds that enter preclinical testing ever make it into clinical trials, with only 20% of these achieving marketing approval. It is therefore very important to advance approaches to decrease the time, reduce costs and improve success rates during the development of drugs.

One approach is the repurposing of high-quality pharmaceutical industry compounds that have stalled at some stage during research or development. Many of these compounds have already undergone preliminary testing in humans, but have not been progressed further because they were not found to be sufficiently effective in the indication for which they were originally developed.

These compounds represent valuable tools that researchers can use to test their novel hypotheses for alternative therapeutic indications, with the ultimate aim of identifying alternative uses for these compounds in other indications. Since partial preclinical and clinical documentation packages have been developed for these assets, any positive findings hold the opportunity to progress towards the market more quickly and cost-effectively, with the ultimate goal of benefiting patients in diseases of high unmet need.

This asset-sharing repurposing programme through IMI2 JU aims to provide researchers across the EU to form hypothesis, to engage in collaborative research with industry and to access discontinued compounds that have already passed through several stages of the drug development process.

In this call, compounds, together with key information on their mechanism of action, pharmacology, safety, tolerability and exclusions, are made available for exploration in the area of Neurodegenerative diseases (please find more details in the topic text). More precisely, applicants are invited to submit proposals to utilise these assets to test their hypotheses, to generate clinical data and, if needed, prerequisite preclinical data, with the ultimate aim of taking these assets to the market in alternative indications to those that they were originally developed for.

Scope:

The overall objective of this pilot programme is to take one of the previously deprioritised clinical compounds and investigate their therapeutic potential in new clinical indications in the area of Neurodegenerative diseases.

These compounds are listed in the topic text together with key information including mechanism of action, original indication, route of administration pharmacology, safety, tolerability and links to previous clinical studies and publications, to facilitate idea generation by investigators with hypotheses for novel uses. These compounds have all been through clinical phase 1 studies.

Proposals should cover clinical Phase 2A proof of concept studies, though larger Phase 2 studies are also in scope if these are within the budget. Clinical submissions should aim at moving towards the next stage of development and positive data should be a starting point for further investment into developing a drug towards clinic and regulatory approval;

If preclinical work is deemed necessary to provide additional support and confidence before moving into a clinical study in an alternative indication, proof-of-concept/feasibility preclinical studies of up to a year in duration can be included in the proposal. These studies should have clear go/no-go criteria for progressing in to the clinical phase of the project.

This programme intends to support only innovative clinical development for the compounds. This means that proposals for clinical development should not be considered in an indication which has been already tested (i.e. original primary indication or additional studies) or if there are already ongoing or planned clinical studies on identical or related disease indications with the compound or with a compound with overlapping mechanism of action that impacts the novelty of a given proposal.

Information on original primary indications, already tested indications, ongoing and/or planned clinical studies for each of these ten compounds can be found in the topic text.

Expected Impact:

Achieving early proof-of-concept for new mechanisms with the potential to rapidly bring novel drugs to patients in areas of high unmet need and/or those with greatest disease burden;

Generation of ideas and/or data licensed from the research organisation, leading to further development of the compound in the new indication;

Added value by repurposing pharmaceutical assets which have already passed through several stages of the R&D process. This can offer significant time, cost and risk savings over embarking on discovery programmes with novel targets;

Supporting EU academic institutions to conduct well-designed and high standard translational and drug development research with quality compounds under GCP conditions, resulting in high impact publications and patents when possible;

Pooling of resources and greater collaboration between the public and private sectors, with the potential for pharmaceutical involvement or establishment of SMEs following in/out-licencing;

Boosting the discovery and development of therapeutics in the area of Neurodegenerative diseases using a more cost-effective approach to drug development;

Advancing science and knowledge of disease (patho)physiology through testing of new hypothesis;

Boosting European competiveness by contributing to the establishment of closer links between industry and academia across the EU, and ensuring Europe is competitive in line with initiatives already in place in other leading scientific regions around the world

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